Pharming Group provides update on ongoing regulatory review of leniolisib for the treatment of APDS in the European Union

Leiden, the Netherlands, May 30, 2024: Pharming Group N.V. (“Pharming” or “the Company”) (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) announces today an update on the ongoing review of its Marketing Authorisation Application (MAA) for leniolisib for the treatment of adult and pediatric patients 12 years of age and older with activated phosphoinositide 3-kinase delta syndrome (APDS) by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA).

Following the May 27-30 CHMP meeting, Pharming received an updated List of Outstanding Issues (LoOI) from the CHMP. The LoOI affirmed the positive clinical benefit and safety of leniolisib, in agreement with the assessment by the Ad Hoc Expert Group (AEG), and included one remaining chemistry, manufacturing and controls (CMC) request.

The CMC request relates to the definition of regulatory starting materials used in the manufacturing process for leniolisib. As Pharming is committed to meeting all of the CHMP’s specific requirements, additional data and quality controls were provided and Pharming proposed implementation of the CMC request post-approval. The CHMP requested that this work be completed pre-approval and has granted Pharming an extension to January 2026 to submit a response. Pharming has already initiated the manufacturing activities requested by the CHMP, which it plans to complete prior to this deadline.

Sijmen de Vries, MD, Chief Executive Officer of Pharming, commented:

“While we are understandably disappointed by the delay to the European license, we are pleased that the CHMP determined the clinical benefit of leniolisib to be positive. The efficacy and safety demonstrated in clinical trials and ongoing real-world experience, with over 300 patient-years of treatment, support that leniolisib fills an unmet medical need. We will continue to work closely with the EMA and CHMP to obtain approval for leniolisib in Europe for people living with APDS. In the meantime, all our clinical development and early access programs will continue.”

The MAA for leniolisib was based on findings from a multinational, triple-blind, placebo-controlled, randomized Phase II/III clinical trial, which met both its co-primary endpoints. The trial evaluated efficacy and safety in 31 patients diagnosed with APDS aged 12 years and older. Also submitted as part of the application were data from a long-term, open-label extension clinical trial in which 37 patients received leniolisib for a median of three years.

Leniolisib is currently available commercially in the United States. The US Food and Drug Administration (FDA) approved leniolisib in March 2023, based on its assessment that leniolisib met clinical and manufacturing standards.

Pharming is maintaining its previously provided 2024 total revenue guidance.

About Activated Phosphoinositide 3-Kinase δ Syndrome (APDS) 

APDS is a rare primary immunodeficiency that was first characterized in 2013. APDS is caused by variants in either one of two identified genes known as PIK3CD or PIK3R1, which are vital to the development and function of immune cells in the body. Variants of these genes lead to hyperactivity of the PI3Kδ (phosphoinositide 3-kinase delta) pathway, which causes immune cells to fail to mature and function properly, leading to immunodeficiency and dysregulation^1,2,3 APDS is characterized by a variety of symptoms, including severe, recurrent sinopulmonary infections, lymphoproliferation, autoimmunity, and enteropathy.^4,5 Because these symptoms can be associated with a variety of conditions, including other primary immunodeficiencies, it has been reported that people with APDS are frequently misdiagnosed and suffer a median 7-year diagnostic delay.⁶ As APDS is a progressive disease, this delay may lead to an accumulation of damage over time, including permanent lung damage and lymphoma.^4-7 A definitive diagnosis can be made through genetic testing. APDS affects approximately 1 to 2 people per million worldwide.

About leniolisib

Leniolisib is an oral small molecule phosphoinositide 3-kinase delta (PI3Kẟ) inhibitor approved in the US and Israel as the first and only targeted treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adult and pediatric patients 12 years of age and older. Leniolisib inhibits the production of phosphatidylinositol-3-4-5-trisphosphate, which serves as an important cellular messenger and regulates a multitude of cell functions such as proliferation, differentiation, cytokine production, cell survival, angiogenesis, and metabolism. Results from a randomized, placebo-controlled Phase II/III clinical trial demonstrated clinical efficacy of leniolisib in the coprimary endpoints; demonstrating statistically significant impact on immune dysregulation and normalization of immunophenotype within these patients, and interim open label extension data has supported the safety and tolerability of long-term leniolisib administration.^8,9 Leniolisib is currently under regulatory review in the European Economic Area, the U.K., Canada and Australia, with plans to pursue further regulatory approvals in Japan and South Korea. Leniolisib is also being evaluated in two Phase III clinical trials in children with APDS.

About Pharming Group N.V.  

Pharming Group N.V. (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) is a global biopharmaceutical company dedicated to transforming the lives of patients with rare, debilitating, and life-threatening diseases. Pharming is commercializing and developing an innovative portfolio of protein replacement therapies and precision medicines, including small molecules and biologics. Pharming is headquartered in Leiden, the Netherlands, and has employees around the globe who serve patients in over 30 markets in North America, Europe, the Middle East, Africa, and Asia-Pacific.

For more information, visit and find us on .  

Forward-Looking Statements   

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References

1. Lucas CL, et al. Nat Immunol. 2014;15(1):88-97.
2. Elkaim E, et al. J Allergy Clin Immunol. 2016;138(1):210-218.
3. Nunes-Santos C, Uzel G, Rosenzweig SD. J Allergy Clin Immunol. 2019;143(5):1676-1687.
4. Coulter TI, et al. J Allergy Clin Immunol. 2017;139(2):597-606.
5. Maccari ME, et al. Front Immunol. 2018;9:543.
6. Jamee M, et al. Clin Rev Allergy Immunol. 2020 Dec;59(3):323-333.
7. Condliffe AM, Chandra A. Front Immunol. 2018;9:338.
8. Rao VK, et al Blood. 2023 Mar 2;141(9):971-983.
9. Rao VK, et al. J Allergy Clin Immunol 2024;153:265-74.
   
   
   
   

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